14 February 2017 EMA/COMP/50377/2017 Procedure Management and Committees Support Division

Committee for Orphan Medicinal Products (COMP) Draft agenda for the meeting on 14-16 February 2017

Chair: Bruno Sepodes – Vice-Chair: Lesley Greene 14 February 2017, 09:00-19:00, room 2F 15 February 2017, 09:00-19:00, room 2F 16 February 2017, 09:00-12:00, room 2F

Health and safety information In accordance with the Agency’s health and safety policy, delegates are to be briefed on health, safety and emergency information and procedures prior to the start of the meeting. Disclaimers Some of the information contained in this agenda is considered commercially confidential or sensitive and therefore not disclosed. With regard to intended therapeutic indications or procedure scopes listed against products, it must be noted that these may not reflect the full wording proposed by applicants and may also vary during the course of the review. Additional details on some of these procedures will be published in the COMP meeting reports once the procedures are finalised. Of note, this agenda is a working document primarily designed for COMP members and the work the Committee undertakes. Note on access to documents Some documents mentioned in the agenda cannot be released at present following a request for access to documents within the framework of Regulation (EC) No 1049/2001 as they are subject to ongoing procedures for which a final decision has not yet been adopted. They will become public when adopted or considered public according to the principles stated in the Agency policy on access to documents (EMA/127362/2006).

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© European Medicines Agency, 2017. Reproduction is authorised provided the source is acknowledged.

Table of contents 1.

Introduction

1.1.

Welcome and declarations of interest of members and experts .............................. 6

1.2.

Adoption of agenda................................................................................................. 6

1.3.

Adoption of the minutes ......................................................................................... 6

2.

Applications for orphan medicinal product designation

2.1.

For opinion ............................................................................................................. 6

2.1.1.

- EMA/OD/285/16 ...................................................................................................... 6

2.1.2.

- EMA/OD/276/16 ...................................................................................................... 8

2.1.3.

- EMA/OD/217/16 ...................................................................................................... 8

2.1.4.

- EMA/OD/219/16 ...................................................................................................... 9

2.1.5.

- EMA/OD/262/16 ...................................................................................................... 9

2.1.6.

- EMA/OD/227/16 .................................................................................................... 10

2.1.7.

- EMA/OD/275/16 .................................................................................................... 11

2.1.8.

- EMA/OD/261/16 .................................................................................................... 11

2.1.9.

- EMA/OD/267/16 .................................................................................................... 11

2.1.10.

- EMA/OD/280/16 .................................................................................................... 12

2.1.11.

- EMA/OD/289/16 .................................................................................................... 12

2.1.12.

- EMA/OD/255/16 .................................................................................................... 13

2.1.13.

- EMA/OD/278/16 .................................................................................................... 13

2.1.14.

- EMA/OD/266/16 .................................................................................................... 13

2.1.15.

- EMA/OD/288/16 .................................................................................................... 13

2.1.16.

- EMA/OD/284/16 .................................................................................................... 14

2.1.17.

- EMA/OD/234/16 .................................................................................................... 14

2.1.18.

- EMA/OD/236/16 .................................................................................................... 14

2.2.

For discussion / preparation for an opinion .......................................................... 15

2.2.1.

- EMA/OD/201/16 .................................................................................................... 15

2.2.2.

- EMA/OD/308/16 .................................................................................................... 15

2.2.3.

- EMA/OD/314/16 .................................................................................................... 16

2.2.4.

- EMA/OD/299/16 .................................................................................................... 17

2.2.5.

- EMA/OD/277/16 .................................................................................................... 17

2.2.6.

- EMA/OD/310/16 .................................................................................................... 17

2.2.7.

- EMA/OD/265/16 .................................................................................................... 18

2.2.8.

- EMA/OD/257/16 .................................................................................................... 18

2.2.9.

- EMA/OD/302/16 .................................................................................................... 18

2.2.10.

- EMA/OD/204/16 .................................................................................................... 18

2.2.11.

- EMA/OD/313/16 .................................................................................................... 19

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2.2.12.

- EMA/OD/270/16 .................................................................................................... 19

2.2.13.

- EMA/OD/315/16 .................................................................................................... 19

2.2.14.

- EMA/OD/309/16 .................................................................................................... 20

2.2.15.

- EMA/OD/287/16 .................................................................................................... 20

2.2.16.

- EMA/OD/301/16 .................................................................................................... 20

2.2.17.

- EMA/OD/294/16 .................................................................................................... 20

2.2.18.

- EMA/OD/293/16 .................................................................................................... 20

2.2.19.

- EMA/OD/303/16 .................................................................................................... 21

2.2.20.

- EMA/OD/300/16 .................................................................................................... 21

2.2.21.

- EMA/OD/286/16 .................................................................................................... 21

2.2.22.

- EMA/OD/253/16 .................................................................................................... 21

2.3.

Revision of the COMP opinions ............................................................................. 22

2.4.

Amendment of existing orphan designations ........................................................ 22

2.5.

Appeal .................................................................................................................. 22

2.5.1.

20% I.V. fat emulsion consisting of 20% soybean oil, 1.2% egg yolk phospholipids, 2.25% glycerin, and water for injection - EMA/OD/062/16 ...................................................... 22

2.6.

Nominations ......................................................................................................... 22

2.6.1.

New applications for orphan medicinal product designation - Appointment of COMP coordinators............................................................................................................ 22

2.7.

Evaluation on-going .............................................................................................. 22

3.

Requests for protocol assistance with significant benefit question 23

3.1.

Ongoing procedures ............................................................................................. 23

3.1.1.

- ............................................................................................................................ 23

3.1.2.

- ............................................................................................................................ 23

3.1.3.

- ............................................................................................................................ 23

3.1.4.

- ............................................................................................................................ 23

3.1.5.

- ............................................................................................................................ 23

3.1.6.

- ............................................................................................................................ 23

3.1.7.

- ............................................................................................................................ 23

3.2.

Finalised letters .................................................................................................... 23

3.2.1.

- ............................................................................................................................ 23

3.2.2.

- ............................................................................................................................ 24

3.3.

New requests........................................................................................................ 24

3.3.1.

- ............................................................................................................................ 24

4.

Review of orphan designation for orphan medicinal products for marketing authorisation 24

4.1.

Orphan designated products for which CHMP opinions have been adopted .......... 24

4.2.

Orphan designated products for discussion prior to adoption of CHMP opinion .... 24

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4.2.1.

- cerliponase alfa - EMA/OD/177/12, EU/3/13/1118, EMEA/H/C/004065 ......................... 24

4.2.2.

- parathyroid hormone – EMA/OD/102/13, EU/3/13/1210, EMEA/H/C/003861 ................ 24

4.2.3.

- pentosan polysulfate sodium – EMA/OD/179/14, EU/3/14/1411, EMEA/H/C/004246 ...... 24

4.3.

Appeal .................................................................................................................. 25

4.4.

On-going procedures ............................................................................................ 25

4.5.

Public Summary of Opinions ................................................................................. 25

5.

Review of orphan designation for authorised orphan medicinal products at time marketing authorisation extension

5.1.

After adoption of CHMP opinion ............................................................................ 25

5.2.

Prior to adoption of CHMP opinion ........................................................................ 25

5.3.

Appeal .................................................................................................................. 25

6.

Application of Article 8(2) of the Orphan Regulation

25

7.

Organisational, regulatory and methodological matters

25

7.1.

Mandate and organisation of the COMP ................................................................ 25

7.1.1.

Protocol Assistance Working Group ............................................................................ 25

7.1.2.

Strategic Review & Learning meetings ........................................................................ 25

7.1.3.

Survey to Committee members, alternates and concerned NCA staff on the service and support provided by EMA Committee Secretariats - Findings ......................................... 26

7.1.4.

Selection procedure for COMP members nominated by the EC on EMA recommendation ... 26

7.2.

Coordination with EMA Scientific Committees or CMDh-v ..................................... 26

7.2.1.

PDCO/COMP Working Group ...................................................................................... 26

7.2.2.

Recommendations on eligibility to PRIME – report from CHMP ....................................... 26

7.3.

Coordination with EMA Working Parties/Working Groups/Drafting Groups ......... 26

7.4.

Cooperation within the EU regulatory network ..................................................... 26

7.4.1.

European Commission .............................................................................................. 26

7.5.

Cooperation with International Regulators........................................................... 26

7.5.1.

Food and Drug Administration (FDA) .......................................................................... 26

7.5.2.

Japanese Pharmaceuticals and Medical Devices Agency (PMDA) ..................................... 26

7.5.3.

The Therapeutic Goods Administration (TGA), Australia ................................................ 26

7.5.4.

Health Canada......................................................................................................... 27

7.6.

Contacts of the COMP with external parties and interaction with the Interested Parties to the Committee ...................................................................................... 27

7.7.

COMP work plan ................................................................................................... 27

7.7.1.

COMP Work Plan 2017 .............................................................................................. 27

7.8.

Planning and reporting ......................................................................................... 27

7.8.1.

List of all applications submitted/expected and the COMP coordinatorship distribution of valid applications submitted in 2017 .......................................................................... 27

7.8.2.

Overview of orphan marketing authorisations/applications ............................................ 27

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8.

Any other business

8.1.

- ............................................................................................................................ 27

9.

Explanatory notes

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1.

Introduction

1.1.

Welcome and declarations of interest of members and experts Pre-meeting list of participants and restrictions in relation to declarations of interests applicable to the items of the agenda for the COMP plenary session to be held 14-16 February 2017. See February 2017 COMP minutes (to be published post March 2017 COMP meeting).

1.2.

Adoption of agenda COMP agenda for 14-16 February 2017.

1.3.

Adoption of the minutes COMP minutes for 17-19 January 2017.

2.

Applications for orphan medicinal product designation

2.1.

For opinion

2.1.1.

- EMA/OD/285/16 Treatment of acute myeloid leukaemia Action: For adoption, Oral explanation to be held on 14 February 2017 at 09:30 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 49 designations for this condition: EMEA/OD/022/00 Gemtuzumab ozogamicin, EMEA/OD/028/04 Midostaurin, EMEA/OD/056/06 Antisense oligonucleotide 5'd[P-Thio] (CCCTG CTCCC CCCTG GCTCC)-3' (see comments box for cenersen sodium), EMEA/OD/098/04 Tipifarnib, EMEA/OD/094/04 Histamine dihydrochloride, EMEA/OD/066/05 1,2-bis(methylsulphonyl)-1-(2-chloroethyl)-2-[(methylamino)carbonyl]hydrazine, EMEA/OD/100/05 zosuquidar trihydrochloride, EMEA/OD/004/06 Decitabine, EMEA/OD/049/07 5'-O-(trans-9"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine, EMEA/OD/087/07 Recombinant human histone H1.3 and recombinant human N-bis-methistone H1.3, EMEA/OD/085/07 Azacitidine, EMEA/OD/099/07 N- (2-Amino-phenyl)-4-[(4pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide, EMEA/OD/118/07 Ribonucleotide reductase R2 specific phosphorothioate oligonucleotide, EMEA/OD/015/08 Sapacitabine, EMEA/OD/048/08 Daunorubicin (liposomal), EMEA/OD/105/08 N-(5-tert-Butylisoxazol-3-yl)N'-{4-[7-(2-(morpholin-4-yl)ethoxy) imidazo[2,1-b][1,3]benzothiazol-2-yl]phenyl}urea dihydrochloride salt, EMEA/OD/028/09 Tosedostat, EMEA/OD/091/09 1-Cyclopropyl-3-[3-(5morpholin-4-ylmethyl-1H-benzoimidazol-2-yl)-1H-pyrazol-4-yl]-urea, EMEA/OD/147/09 2methoxymethyl-2-hydroxymethyl-1-azabicyclo[2,2,2]octan-3-one, EMA/OD/044/10 Allogeneic T cells encoding an exogenous TK gene, EMA/OD/094/10 N-[(2S)-2,3dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl) amino] isonicotinamide hydrochloride,

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EMA/OD/161/10 Allogeneic bone marrow stem cells treated ex vivo with 16,16-dimethyl prostaglandin E2, EMA/OD/101/11 Allogeneic human dendritic cells derived from a CD34+ progenitor cell line, EMA/OD/070/11 Liposomal combination of cytarabine and daunorubicin, EMA/OD/158/11 Vosaroxin, EMA/OD/167/12 L-asparaginase encapsulated in erythrocytes, EMA/OD/064/13 trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bishydrochloride, EMA/OD/141/13 (2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3(2-(5-(tert-butyl)-1Hbenzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl) amino)methyl)tetrahydrofuran-3,4-diol, EMA/OD/181/13 Volasertib, EMA/OD/100/14 4{[(2R,3S,4R,5S)-4-(4-Chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid, EMA/OD/061/14 (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'(pyrazin-2-yl)acrylohydrazide, EMA/OD/103/14 Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment, EMA/OD/175/14 Allogeneic ex vivogenerated natural killer cells from CD34+ umbilical cord blood progenitor cells, EMA/OD/240/14 Alvocidib, EMA/OD/188/14 Allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells / allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic CD133- cells, EMA/OD/258/14 Ulocuplumab, EMA/OD/045/15 inecalcitol, EMA/OD/037/15 2-((3-((4-((3aminopropyl)amino)butyl)amino)propyl)amino)-N-((5S,5aS,8aR,9R)-9-(4-hydroxy-3,5dimethoxyphenyl)-8-oxo-5,5a,6,8,8a,9-hexahydrofuro[3',4':6,7]naphtho[2,3-d][1,3]dioxol5-yl)acetamide, tetrahydrochloride, EMA/OD/089/15 CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA cross-linking pyrrolobenzodiazepine dimer drug, EMA/OD/112/15 Recombinant human interleukin-3 truncated diphtheria toxin fusion protein, EMA/OD/145/15 Humanised monoclonal antibody of the IgG4 kappa isotype targeting CD47, EMA/OD/165/15 Sodium (2R,3S,5R)-5-(4amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate, EMA/OD/144/15 Combretastatin A1-diphosphate, EMA/OD/180/15 Arsenic trioxide, EMA/OD/205/15 Venetoclax, EMA/OD/233/15 Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, SerGly-Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-AspGlu-Leu-Val-Arg-His-His-Asn-Met-His-Gln-Arg-Asn-Met-Thr-Lys-Leu and Pro-Gly-Cys-AsnLys-Arg-Tyr-Phe-Lys-Leu-Ser-His-Leu-Gln-Met-His-Ser-Arg-Lys-His-Thr-Gly, EMA/OD/253/15 2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate, EMA/OD/155/16 P-ethoxy growth factor receptor-bound protein 2 (Grb2) antisense oligonucleotide, EMA/OD/197/16 Ivosidenib Designations withdrawn: EMEA/OD/065/02 2-chloro-9-[2-deoxy-2-fluoro-ß-Darabinofuranosyl]adenine, EMEA/OD/051/04 Homoharringtonine, EMEA/OD/059/04 Val-LeuGln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3), EMEA/OD/045/05 Troxacitabine, EMEA/OD/018/06 Human monoclonal antibody against inhibitory killer cell lg-like receptors (1-7 F9), EMEA/OD/020/06 Lestaurtinib, EMEA/OD/024/07 Arsenic trioxide, EMEA/OD/069/07 Amonafide L-malate, EMEA/OD/060/08 2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate, EMEA/OD/118/08 Lintuzumab, EMEA/OD/090/08 Allogeneic ex vivo expanded umbilical cord blood cells, EMEA/OD/016/09 26 base single stranded phosphodiester DNA oligonucleotide, EMEA/OD/132/09 (1S, 2S, 3R, 4R)-3-(5-Fluoro-2-(3-methyl-4-(4-methylpiperazin-1-yl)phenylamino)-pyrimidin-4-ylamino)-bicyclo[2.2.1]hept-5-ene-2-carboxamide benzoate),

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EMA/OD/023/10 1-[2-(Benzo[1,2,5]thiadiazol-5-ylamino)-6-(2,6-dichloro-phenyl)pyrido[2,3-d]pyrimidin-7-yl]-3-tert-butyl-urea, EMA/OD/156/10 Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2, EMA/OD/067/11 1-(4-{4amino-7-[1-(2-hydroxyethyl)-1H- pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl}phenyl)-3-(3fluorophenyl)urea, EMA/OD/105/12 Liposomal daunorubicin

2.1.2.

- EMA/OD/276/16 Treatment of Niemann-Pick disease type C Action: For adoption, Oral explanation to be held on 14 February 2017 at 11:00 Documents tabled: Draft Summary report with response to LoQs

2.1.3.

- EMA/OD/217/16 Treatment of cystic fibrosis Action: For adoption, Oral explanation to be held on 14 February 2017 at 14:30 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 37 designations for this condition: EMEA/OD/032/00 L-Lysine-N-Acetyl-LCysteinate, EMEA/OD/011/03 Recombinant dog gastric lipase, EMEA/OD/038/02 Duramycin, EMEA/OD/039/04 Dexamethasone sodium phosphate encapsulated in human erythrocytes, EMEA/OD/053/04 Alpha-1 antitrypsin (inhalation use), EMEA/OD/107/04 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid, EMEA/OD/062/05 Mannitolum, EMEA/OD/001/06 Heparin sodium, EMEA/OD/037/09 Ciprofloxacin (liposomal), EMEA/OD/092/06 Ciprofloxacin (inhalation use), EMEA/OD/104/06 Alginate oligosaccharide (G-block) fragment, EMEA/OD/041/07 Alpha1-proteinase inhibitor (inhalation use), EMEA/OD/031/08 Avian polyclonal IgY antibody against Pseudomonas aeruginosa, EMEA/OD/010/08 N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3carboxamide, EMEA/OD/009/09 Hypothiocyanite / lactoferrin, EMA/OD/040/10 Nafamostat mesilate, EMA/OD/024/10 3-(6-(1-(2,2-difluorobenzo [d] [1,3] dioxol-5yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid, EMA/OD/032/11 Sinapultide, dipalmitoylphosphatidylcholine palmitoyl-oleoyl phosphatidylglycerol, sodium salt and palmitic acid, EMA/OD/037/11 Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phospatidylinositol and cholesterol, EMA/OD/046/11 Cysteamine, EMA/OD/058/12 Alpha-1 proteinase inhibitor (for inhalation use), EMA/OD/005/13 Recombinant human CXCL8 mutant, EMA/OD/017/13 4,6,4’-trymethylangelicin, EMA/OD/096/13 Antisense oligonucleotide targeting the F508delta mutation of CFTR, EMA/OD/095/13 Nitric oxide, EMA/OD/159/13 Cysteamine, EMA/OD/156/13 11-(4-Dimethylamino-3-hydroxy-6-methyltetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa6-aza-cyclopentadecane-13,15-dione, EMA/OD/036/14 Nitric oxide, EMA/OD/013/14 Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent, EMA/OD/002/14 1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1hydroxy-2-methylpropan-2-yl)-1Hindol-5-yl}cyclopropanecarboxamide, EMA/OD/131/14 4-

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[[(1S,4S)-5-[[4-[4-(Oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2yl]methyl]benzoic acid, EMA/OD/018/15 2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl)acetamide, EMA/OD/319/14 Nitric oxide, EMA/OD/068/15 Fixed-dose combination of fosfomycin disodium and tobramycin, EMA/OD/061/15 Recombinant human acid ceramidase, EMA/OD/013/16 Sodium nitrite and ethylenediaminetetraacetic acid, EMA/OD/100/16 (6aR, 10aR)-3-(1’,1’-dimethylheptyl)delta-8-tetrahydro-cannabinol-9-carboxylic acid Designations withdrawn: EMEA/OD/009/02 Carbamic acid/[[4-[[3-[[4-[1-(4hydroxyphenyl)-1-methyl-ethyl]phenoxy]methyl]phenyl]methoxy]-phenyl]iminomethyl],ethyl ester, EMEA/OD/064/00 8-cyclopentyl-1, 3-dipropylxanthine, EMEA/OD/018/03 Engineered protein inhibitor of human neutrophil elastase, EMEA/OD/075/02 Amiloride hydrochloride dihydrate, EMEA/OD/023/04 Recombinant human bile salt-stimulated lipase, EMEA/OD/054/05 Heparin sodium (inhalation use), EMEA/OD/072/05 Denufosol tetrasodium, EMEA/OD/118/05 Glutathione, EMEA/OD/024/08 Levofloxacin hemihydrate, EMA/OD/032/14 Lumacaftor/ivacaftor

2.1.4.

- EMA/OD/219/16 Treatment of invasive aspergillosis Action: For adoption, Oral explanation to be held on 14 February 2017 at 14:30 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 2 designations for this condition: EMA/OD/009/14 Isavuconazonium sulfate, EMA/OD/104/16 2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)- N-{4-[4-(5-fluoropyrimidin-2-yl) piperazin- 1-yl]-phenyl}-2-oxo-acetamide

2.1.5.

- EMA/OD/262/16 Treatment of pancreatic cancer Action: For adoption, Oral explanation to be held on 14 February 2017 at 17:00 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 35 designations for this condition: EMEA/OD/055/02 G17(9) gastrinDiphtheria Toxoid Conjugate, EMEA/OD/068/02 Rubitecan, EMEA/OD/009/05 Bovine bile extract, EMEA/OD/028/05 4-imino-1, 3-diazobicyclo-[3.1.0]-hexan-2-one, EMEA/OD/063/06 Paclitaxel (liposomal), EMEA/OD/026/06 Human telomerase reverse transcriptase peptide (611-626), EMEA/OD/103/06 Cisplatin (liposomal), EMEA/OD/100/08 L-asparaginase encapsulated in erythrocytes, EMEA/OD/006/08 Nimotuzumab, EMEA/OD/080/08 Yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1, EMEA/OD/101/08 S-[2,3-bispalmitoyloxy-(2R)-propyl]-cysteinyl-GNNDESNISFKEK, EMEA/OD/030/09 Trabedersen, EMEA/OD/105/09 Brivudine, EMEA/OD/069/09 N-[(2S)-2,3dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino]isonicotinamide hydrochloride, EMEA/OD/063/09 Masitinib mesilate, EMA/OD/135/10 Glufosfamide, EMA/OD/150/10 Salirasib, EMA/OD/007/11 Mixture of seven synthetic fragments consisting of p21 RAS

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peptides, EMA/OD/008/11 Genetically modified human adenovirus encoding human PH20 hyaluronidase, EMA/OD/051/11 Nanoliposomal irinotecan, EMA/OD/065/12 Mixture of two allogeneic human pancreatic cancer cell lines stably transduced with a retroviral vector encoding the murine alpha-(1,3)-galactosyltransferase gene, EMA/OD/037/13 (1-methyl-2nitro-1H-imidazole-5-yl)methyl N,N’-bis(2-bromoethyl)diamidophosphate, EMA/OD/071/13 Chimeric monoclonal antibody against claudin-18 splice variant 2, EMA/OD/164/13 Cysteamine bitartrate, EMA/OD/081/14 Immunoglobulin G1 (TEXT TOO LONG), EMA/OD/085/14 [5-Amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxypropoxy)-phenyl]-methanone, EMA/OD/187/14 Herpes simplex type 1 virus containing cellular B-myb gene as tumour-specific promoter, EMA/OD/143/14 Heat-killed Mycobacterium obuense (whole cell), EMA/OD/173/14 Pegylated recombinant human hyaluronidase PH20, EMA/OD/302/14 Human reovirus type 3 Dearing strain, EMA/OD/034/15 Modified adenovirus serotype 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand (TMZCD40L) and full length 4-1BBL, EMA/OD/168/15 Live attenuated Listeria monocytogenes delta actA/delta inlB strain expressing human mesothelin, EMA/OD/169/15 Two allogenic irradiated pancreatic tumour cell lines, EMA/OD/193/16 Pegylated recombinant human interleukin-10, EMA/OD/241/16 Antroquinonol Designations withdrawn: EMEA/OD/070/02 Iodine (131I) Anti-CEA sheep-human chimeric monoclonal antibody, EMEA/OD/040/04 Deuterium oxide, EMEA/OD/097/05 26 base single stranded phosphodiester DNA oligonucleotide, EMEA/OD/111/07 Chimeric antibody to mesothelin, EMEA/OD/067/09 5'-O-(trans-9''-octadecenoyl)-1-beta-D-2’deoxy-2’,2’difluorocytidine, EMA/OD/087/10 Nanoparticle albumin-bound paclitaxel, EMA/OD/007/12 Polyinosine-polycytidylic acid coupled with the polycationic polyethyleneimine, EMA/OD/145/12 Recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem cell antigen

2.1.6.

- EMA/OD/227/16 Treatment of epidermolysis bullosa Action: For adoption, Oral explanation to be held on 14 February 2017 at 18:00 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 11 designations for this condition: EMEA/OD/111/05 Bilayer engineered skin composed of keratinocytes from the patient (autologous) and fibroblasts from a donor (allogeneic) embedded in a plasma matrix, EMEA/OD/061/09 Allogeneic human dermal fibroblasts, EMA/OD/120/10 Dry extract from birch bark (DER 0.1-0.2:1), extraction solvent n-heptane 95% (V/V), EMA/OD/145/13 Allantoin, EMA/OD/149/13 Diacerein, EMA/OD/201/13 Recombinant human alpha 1 chain homotrimer of type VII collagen, EMA/OD/197/14 Allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis, EMA/OD/218/15 Autologous dermal fibroblasts genetically modified ex vivo with a lentiviral vector containing the human COL7A1 gene, EMA/OD/299/14 Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a COL17A1-encoding retroviral vector, EMA/OD/297/14 Ex-vivoexpanded autologous human keratinocytes containing epidermal stem cells transduced with

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a LAMB3-encoding retroviral vector, EMA/OD/188/15 Ex-vivo-expanded autologous fibroblasts transduced with lentiviral vector containing the COL7A1 gene Designation withdrawn: EMA/OD/172/10 Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh

2.1.7.

- EMA/OD/275/16 Treatment of Lennox-Gastaut syndrome Action: For adoption, Oral explanation to be held on 15 February 2017 at 09:00 Documents tabled: Draft Summary report with response to LoQs Notes: There has been 1 designation for this condition: EMEA/OD/047/04 Rufinamide

2.1.8.

- EMA/OD/261/16 Treatment of idiopathic pulmonary fibrosis Action: For adoption, Oral explanation to be held on 15 February 2017 at 10:00 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 12 designations for this condition: EMEA/OD/052/04 Pirfenidone, EMEA/OD/054/07 Interferon gamma, EMEA/OD/104/09 Macitentan, EMA/OD/079/10 2-(2chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine3,6(2H,5H)-dione, EMA/OD/091/11 4-[[9-[(3S)-tetrahydro-3-furanyl]-8-[(2,4,6trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol, EMA/OD/048/12 Recombinant human pentraxin-2, EMA/OD/186/12 nintedanib, EMA/OD/051/14 Humanised anti-alpha ν beta 6 monoclonal antibody, EMA/OD/130/14 1-(6-benzothiazolylsulfonyl)-5chloro-1H-indole-2-butanoic acid, EMA/OD/072/15 3-pentylbenzeneacetic acid sodium salt, EMA/OD/046/16 3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(Nbutyloxylcarbamate)-sulphonamide] sodium salt, EMA/OD/088/16 2-((2-ethyl-6-(4-(2-(3hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile Designations withdrawn: EMEA/OD/002/05 Interferon gamma, EMEA/OD/033/04 HeparinSodium, EMEA/OD/075/04 Acetylcysteine, EMEA/OD/105/07 Recombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3, EMEA/OD/027/08 Bosentan, EMA/OD/029/10 Ambrisentan, EMA/OD/111/12 Tralokinumab

2.1.9.

- EMA/OD/267/16 Treatment of Neuromyelitis Optica Spectrum Disorders (NMOSD) Action: For adoption, Oral explanation to be held on 15 February 2017 at 12:00 Documents tabled: Draft Summary report with response to LoQs

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2.1.10.

- EMA/OD/280/16 Treatment of retinitis pigmentosa Action: For adoption, Oral explanation to be held on 15 February 2017 at 14:30 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 20 designations for this condition: EMEA/OD/057/06 4,7,10,13,16,19Docosahexaenoic acid, EMEA/OD/043/07 Adenovirus associated viral vector serotype 4 containing the human RPE65 gene, EMEA/OD/087/08 Recombinant human proinsulin, EMA/OD/162/10 9-cis-Retinyl acetate, EMA/OD/159/11 Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor, EMA/OD/006/12 Recombinant human methionine proinsulin, EMA/OD/025/13 Expanded human allogeneic neural retinal progenitor cells extracted from neural retina, EMA/OD/015/13 Recombinant human nerve growth factor, EMA/OD/031/13 Adenovirus associated viral vector serotype 5 containing the human pde6β gene, EMA/OD/289/14 Sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7atetrahydro-4H-furo[3,2-d][1,3,2]dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2a]purin-9-one, EMA/OD/271/14 Myriocin, EMA/OD/327/14 Recombinant human mesencephalic astrocyte-derived neurotrophic factor, EMA/OD/040/15 Adenovirusassociated viral vector serotype 2 containing the human RPE65 gene, EMA/OD/158/16 Adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein, EMA/OD/213/15 Allogeneic fetal human retinal progenitor cells expanded ex vivo, EMA/OD/208/15 4-[(2E)-1-oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2propen-1-yl]-1-piperazinecarboxamide, EMA/OD/028/16 Adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene, EMA/OD/102/16 Adenovirus associated viral vector serotype 5 containing the human RPGR gene, EMA/OD/146/16 Adeno-associated viral vector serotype 5 containing the human RLBP1 gene, EMA/OD/165/16 Adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes Designations withdrawn: EMEA/OD/075/07 Recombinant human rod-derived cone viability factor, EMEA/OD/106/07 Allogeneic human umbilical cord tissue-derived cells, EMA/OD/021/12 17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin (after administration of adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5), EMA/OD/135/12 Adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 (prior to administration of 17dimethylaminoethylamino-17-demethocygeldanamycin), EMA/OD/067/13 Unoprostone isopropyl

2.1.11.

- EMA/OD/289/16 Treatment of cystic fibrosis Action: For information Documents tabled: Withdrawal request of 30 January 2017 Notes:

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Withdrawn There have been 37 designations for this condition: Please see 2.1.3.

2.1.12.

- EMA/OD/255/16 Treatment of pancreatic cancer Action: For adoption, Oral explanation to be held on 15 February 2017 at 17:00 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 35 designations for this condition: Please see 2.1.5.

2.1.13.

- EMA/OD/278/16 Treatment of short bowel syndrome Action: For information Documents tabled: Withdrawal request of 27 January 2017 Notes: Withdrawn There have been 3 designations for this condition: EMEA/OD/045/01 [gly2]-recombinant human glucagon-like peptide, EMA/OD/080/14 Oxalobacter formigenes strain HC-1, EMA/OD/050/15 Insulin human (rDNA)

2.1.14.

- EMA/OD/266/16 Treatment of pancreatic cancer Action: For adoption, Oral explanation to be held on 16 February 2017 at 09:00 Documents tabled: Draft Summary report with response to LoQs Notes: There have been 35 designations for this condition: Please see 2.1.5.

2.1.15.

- EMA/OD/288/16 Treatment of short bowel syndrome Action: For information Documents tabled: Withdrawal request of 31 January 2017 Notes: Withdrawn

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There have been 3 designations for this condition: EMEA/OD/045/01 [gly2]-recombinant human glucagon-like peptide, EMA/OD/080/14 Oxalobacter formigenes strain HC-1, EMA/OD/050/15 Insulin human (rDNA)

2.1.16.

- EMA/OD/284/16 Treatment of amyotrophic lateral sclerosis Action: For information Documents tabled: Withdrawal request of 31 January 2017 Notes: Withdrawn There have been 19 designations for this condition: EMEA/OD/053/06 Arimoclomol, EMEA/OD/102/07 Filgrastim, EMEA/OD/096/08 (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6benzothiazole-diamine dihydrochloride monohydrate, EMEA/OD/108/09 Recombinant human vascular endothelial growth factor, EMA/OD/043/11 Smilagenin, EMA/OD/106/11 S[+] apomorphine, EMA/OD/138/11 6-ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)one, EMA/OD/011/13 Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors, EMA/OD/023/13 Sodium chlorite, EMA/OD/044/13 Allogeneic motor neuron progenitor cells derived from human embryonic stem cells, EMA/OD/184/14 Edaravone, EMA/OD/283/14 Enoxacin, EMA/OD/032/15 Edaravone, EMA/OD/051/15 Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate, EMA/OD/011/16 H-Phe-Ser-Arg-Tyr-AlaArg-OH-acetate, EMA/OD/241/15 Recombinant human cerebral dopamine neurotrophic factor, EMA/OD/081/16 Masitinib mesilate, EMA/OD/120/16 Synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid, EMA/OD/182/16 Ibudilast Designations withdrawn: EMEA/OD/029/00 Xaliproden hydrochloride, EMEA/OD/030/06 Cholest-4-en-3-one, oxime, EMEA/OD/125/07 Sarsasapogenin, EMEA/OD/012/09 Talampanel, EMA/OD/060/10 Recombinant humanised monoclonal antibody to human Nogo-A protein of the IgG1/kappa class

2.1.17.

- EMA/OD/234/16 Treatment of granulosa cell tumours Action: For adoption Documents tabled: Draft Summary report with response to LoQs

2.1.18.

- EMA/OD/236/16 Treatment of granulosa cell tumours Action: For adoption Documents tabled: Draft Summary report with response to LoQs

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2.2. 2.2.1.

For discussion / preparation for an opinion - EMA/OD/201/16 Treatment of soft tissue sarcoma Action: For adoption Documents tabled: Draft Summary report Notes: There have been 15 designations for this condition: EMEA/OD/001/01 Ecteinascidin 743, EMEA/OD/042/06 Doxorubicin hydrochloride (liposomal), EMA/OD/129/16 Crenolanib besylate, EMA/OD/155/11 Yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10, EMA/OD/041/13 Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor, EMA/OD/190/13 Doxorubicin(6-maleimidocaproyl)hydrazone, EMA/OD/037/16 16-base single-stranded peptide nucleic acid oligonucleotide linked to a 7 aminoacid peptide, EMA/OD/266/14 Olaratumab, EMA/OD/159/15 Glucopyranosyl lipid A stable emulsion and recombinant New York esophageal squamous cell carcinoma-1 protein, EMA/OD/184/15 (S)-N-(5-((R)-2-(2,5difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1carboxamide hydrogen sulfate, EMA/OD/215/15 Human/murine chimeric monoclonal antibody against endoglin, EMA/OD/238/15 Sindbis virus envelope pseudotyped lentiviral vector encoding New York esophageal squamous cell carcinoma-1 protein, EMA/OD/064/16 Autologous CD4+ and CD8+ T cells transduced with lentiviral vector containing an affinityenhanced T-cell receptor targeting the New York esophageal antigen-1, EMA/OD/108/16 A non-covalent trimer of tumor necrosis factor fused to an antibody specific to the extradomain B of fibronectin in single-chain variable fragment format, EMA/OD/166/16 Propranolol Designations withdrawn: EMEA/OD/059/03 N-acetylsarcosyl-glycyl-L-valyl-D-alloisoleucylL-threonyl-L-norvalyl-L-isoleucyl-L-arginyl-L-prolyl-N-ethylamide, EMEA/OD/050/05 (1R, 2R, 4S)-4-{(2R)-2-[(3S,6R,7E,9R,10R,12R,14S,15E,17E,19E,21S,23S,26R, 27R,34aS)9,27-dihydroxy-10,21-dimethoxy-6,8,12,14,20,26-hexamethyl-1,5,11,28,29-pentaoxo1,4,5,6,9,10,11,12,13,14,21,22,23,24,25,26,27,28,29,31,32,33,34,34a-tetra-cosahydro3H-23,27-epoxypyrido[2,1-c][1,4]oxazacyclohentriacontin-3-yl]propyl}-2-methoxycyclohexyldimethyl-phosphinate, EMEA/OD/071/05 Brostallicin, EMEA/OD/083/06 Fenretinide, EMEA/OD/044/08 Palifosfamide, EMA/OD/141/10 Ombrabulin, EMA/OD/110/11 (1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl) diamidophosphate

2.2.2.

- EMA/OD/308/16 Treatment of acromegaly Action: For adoption Documents tabled: Draft Summary report Notes: There have been 5 designations for this condition: EMEA/OD/010/09 Octreotide chloride (lipid depot solution), EMEA/OD/051/09 Pasireotide, EMA/OD/107/12 Cyclo(-gamma-

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aminobutyryl-L-phenylalanyl-L-tryptophanyl-D-tryptophanyl-L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt, EMA/OD/042/13 Octreotide acetate (oral use), EMA/OD/023/16 2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor Designation withdrawn: EMA/OD/108/11 Recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin serotype D

2.2.3.

- EMA/OD/314/16 Treatment of ovarian cancer Action: For adoption Documents tabled: Draft Summary report Notes: There have been 30 designations for this condition: EMEA/OD/019/02 Oregovomab, EMEA/OD/061/06 Paclitaxel (micellar), EMEA/OD/080/03 Anti-epithelial cell adhesion molecule/anti-CD3 monoclonal antibody, EMEA/OD/044/03 Trabectedin, EMEA/OD/065/05 Imexon, EMEA/OD/063/07 Olaparib, EMEA/OD/110/07 Humanised monoclonal antibody to the folate receptor alpha, EMEA/OD/006/09 Human MHC non-restricted cytotoxic T-cell line, EMEA/OD/086/09 8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4f][1,6]naphthyridin-3(2H)-one mono-hydrochloride, EMA/OD/015/10 (3S)-3-{4-[7(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt, EMA/OD/021/10 Autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 - glutathione S transferase) coupled to oxidised polymannose, EMA/OD/014/10 Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt, EMA/OD/111/10 Veliparib, EMA/OD/054/11 20-pentaerythritol poly (oxy-1,2-ethanediyl)carboxymethyl-glycinate-7-ethyl-10-hydroxycamptothecine 10-[1,4'-bipiperidine]-1'carboxylate, EMA/OD/151/11 2-Allyl-1-[6-(1-hydroxy-1-methylethyl)pyridin-2-yl]-6-{[4-(4methylpiperazin-1-yl)phenyl]amino}-1,2-dihydro-3H-pyrazolo[3,4-d]pyrimidin-3-one, EMA/OD/085/12 rucaparib, EMA/OD/099/12 Lurbinectedin, EMA/OD/147/12 Chimeric monoclonal antibody against claudin 6, EMA/OD/039/13 Fosbretabulin tromethamine, EMA/OD/122/13 Trebananib, EMA/OD/186/13 Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor, EMA/OD/059/14 Cediranib, EMA/OD/281/14 Humanised anti-folate receptor 1 monoclonal antibody conjugated to maytansinoid DM4, EMA/OD/157/14 2-hydroxymethyl-2-methoxymethyl-1azabicyclo[2,2,2]octan-3-one, EMA/OD/211/14 Chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 regions, EMA/OD/223/14 N-methyl-4-({4-[({3methyl(methylsulfonyl)amino]pyrazin-2- yl}methyl)amino]-5- (trifluoromethyl)pyrimidin-2yl}amino)benzamide hydrochloride, EMA/OD/304/14 Human reovirus type 3 Dearing strain, EMA/OD/314/14 {2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}N,N-dipropylcarboxamide, EMA/OD/126/15 (5S,8S,10aR)-N-benzhydryl-5-((S)-2(methylamino)propanamido)-3-(3-methylbutanoyl)-6-oxodecahydropyrrolo[1,2a][1,5]diazocine-8-carboxamide, EMA/OD/159/16 Vaccine consisting of 5 survivin peptides with different human leukocyte antigen restrictions Designations withdrawn: EMEA/OD/061/00 Human Milk Fat Globule 1 / Yttrium (90Y) human Milk Fat Globule 1 - S p isothiocyanatobenzyl-diethylenetriaminepentaacetic acid,

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EMEA/OD/062/01 Epothilone B, EMEA/OD/016/03 Murine anti-idiotypic antibody against OC125 antibody against CA125 antigen, EMEA/OD/071/09 Anti-EphA2 monoclonal antibody conjugated to maleimidocaproyl monomethylauristatin phenylalanine, EMA/OD/094/11 Vincaleukoblastin-23-oic acid, O4-deacetyl-2-[(2-mercaptoethoxy)carbonyl]hydrazide, disulfide with …, EMA/OD/002/12 1-(4-{4-amino-7-[1-(2-hydroxyethyl)-1H- pyrazol-4-yl] thieno [3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea, EMA/OD/114/12 Alisertib

2.2.4.

- EMA/OD/299/16 Treatment of pulmonary arterial hypertension Action: For adoption Documents tabled: Draft Summary report Notes: There have been 4 designations for this condition: EMEA/OD/018/08 Beraprost sodium, EMA/OD/023/11 Macitentan, EMA/OD/111/11 Sodium nitrite, EMA/OD/179/15 Ubenimex

2.2.5.

- EMA/OD/277/16 Treatment of Fabry disease Action: For adoption Documents tabled: Draft Summary report Notes: There have been 3 designations for this condition: EMEA/OD/105/05 1deoxygalactonojirimycin hydrochloride, EMA/OD/042/12 N-Butyldeoxygalactonojirimycin, EMA/OD/052/14 (3S)-1-azabicyclo[2.2.2]oct-3-yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4yl]propan-2-yl}carbamate

2.2.6.

- EMA/OD/310/16 Treatment of ornithine transcarbamylase deficiency Action: For adoption Documents tabled: Draft Summary report Notes: There have been 4 designations for this condition: EMEA/OD/101/07 Heterologous human adult liver derived stem cells, EMA/OD/026/11 Heterologous human adult liver-derived stem cells, EMA/OD/227/15 Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase, EMA/OD/053/16 Sodium benzoate Designation withdrawn: EMA/OD/097/11 Sodium phenylbutyrateHuman heterologous liver cells (for infusion)

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2.2.7.

- EMA/OD/265/16 Treatment of GM1 gangliosidosis Action: For adoption Documents tabled: Draft Summary report

2.2.8.

- EMA/OD/257/16 Treatment in haematopoietic stem cell transplantation Action: For adoption Documents tabled: Draft Summary report Notes: There have been 5 designations for this condition: EMA/OD/008/16 Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment, EMA/OD/020/16 Allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19, EMA/OD/090/16 Radio-iodinated (131I) anti-CD45 murine monoclonal antibody, EMA/OD/149/16 Allogeneic peripheral blood mononuclear cells incubated ex vivo with 16, 16-dimethyl prostaglandin E2 and dexamethasone, EMA/OD/191/16 Human donor haematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein fused to MYC transcription factor

2.2.9.

- EMA/OD/302/16 Treatment of epidermolysis bullosa due to mutations in the COL7A1 gene Action: For adoption Documents tabled: Draft Summary report

2.2.10.

- EMA/OD/204/16 Treatment of thromboangiitis obliterans (Buerger's disease) Action: For adoption Documents tabled: Draft Summary report Notes: There have been 2 designations for this condition: EMA/OD/041/10 Autologous bone marrow-derived mononuclear cell fraction, EMA/OD/019/15 Adult human bone-marrowderived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells

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2.2.11.

- EMA/OD/313/16 Treatment of Asherman's syndrome Action: For adoption Documents tabled: Draft Summary report

2.2.12.

- EMA/OD/270/16 Treatment of multiple myeloma Action: For adoption Documents tabled: Draft Summary report Notes: There have been 14 designations for this condition: EMEA/OD/040/01 Thalidomide, EMEA/OD/063/03 3-(4'aminoisoindoline-1'-one)-1-piperidine-2,6-dione, EMEA/OD/044/04 Aplidine, EMEA/OD/066/04 Recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors, EMEA/OD/012/05 N-(methyl-diazacyclohexylmethylbenzamide)-azaphenyl-aminothiopyrrole, EMEA/OD/120/07 Carfilzomib, EMEA/OD/068/08 N2'-Deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]maytansine-chimerized anti-CD138 IgG4 monoclonal antibody, EMEA/OD/076/08 Human anti-intercellular adhesion molecule-1 monoclonal antibody, EMEA/OD/053/08 Milatuzumab, EMEA/OD/053/09 Pomalidomide, EMA/OD/017/11 Acadesine, EMA/OD/048/11 2,2'-{2[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2dioxaborolane-4,4-diyl}diacetic acid, EMA/OD/113/12 Panobinostat, EMA/OD/121/16 Venetoclax Designations withdrawn: EMEA/OD/048/00 Arsenic trioxide, EMEA/OD/003/01 Humanised anti-HM1.24 monoclonal antibody, EMEA/OD/018/00 Thalidomide, EMEA/OD/026/01 Deoxyribose phosphorothioate (5'-tct-ccc-agc-gtg-cgc-cat-3'), EMEA/OD/019/01 Thalidomide, EMEA/OD/070/04 17-allylamino-17-demethoxygeldanamycin, EMEA/OD/093/05 Human monoclonal antibody against HLA-DR, EMEA/OD/003/09 Chimericanti-interleukin-6 monoclonal antibody, EMEA/OD/133/09 Dexamethasone (40 mg tablet), EMEA/OD/130/09 Perifosine, EMA/OD/115/10 Maytansinoid-conjugated humanised monoclonal antibody against CD56, EMA/OD/137/10 Vorinostat, EMA/OD/137/11 Chimeric monoclonal antibody against kappa myeloma antigen, EMA/OD/061/12 Elotuzumab

2.2.13.

- EMA/OD/315/16 Treatment of acute myeloid leukaemia Action: For adoption Documents tabled: Draft Summary report Notes: There have been 49 designations for this condition: Please see 2.1.1.

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2.2.14.

- EMA/OD/309/16 Treatment of neonatal encephalopathy Action: For adoption Documents tabled: Draft Summary report Notes: There has been 1 designation for this condition: EMA/OD/141/16 Autologous mononuclear cells derived from human cord blood

2.2.15.

- EMA/OD/287/16 Treatment of graft rejection following solid organ transplantation Action: For adoption Documents tabled: Draft Summary report

2.2.16.

- EMA/OD/301/16 Treatment of narcolepsy Action: For adoption Documents tabled: Draft Summary report Notes: There have been 3 designations for this condition: EMEA/OD/087/06 1-{3-[3-(4chlorophenyl)propoxy]propyl}piperidine, hydrochloride, EMA/OD/254/14 Mazindol, EMA/OD/002/15 Mazindol Designation withdrawn: EMEA/OD/051/02 Sodium oxybate

2.2.17.

- EMA/OD/294/16 Treatment of calciphylaxis Action: For adoption Documents tabled: Draft Summary report Notes: There have been 3 designations for this condition: EMA/OD/116/10 Sodium thiosulfate, EMA/OD/149/11 Sodium thiosulfate, EMA/OD/043/12 Hexasodium phytate

2.2.18.

- EMA/OD/293/16 Treatment of fragile X syndrome Action: For adoption

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Documents tabled: Draft Summary report Notes: There have been 7 designations for this condition: EMA/OD/144/10 R-baclofen, EMA/OD/059/12 Mavoglurant, EMA/OD/105/14 (3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one, EMA/OD/137/14 Acamprosate calcium, EMA/OD/253/14 Tideglusib, EMA/OD/055/15 Glycyl-L-2-methylprolyl-L-glutamic acid, EMA/OD/034/16 Pyridoxine and L-pyroglutamic acid

2.2.19.

- EMA/OD/303/16 Treatment of cystic fibrosis Action: For adoption Documents tabled: Draft Summary report Notes: There have been 37 designations for this condition: Please see 2.1.3.

2.2.20.

- EMA/OD/300/16 Treatment of ovarian cancer Action: For adoption Documents tabled: Draft Summary report Notes: There have been 30 designations for this condition: Please see 2.2.3.

2.2.21.

- EMA/OD/286/16 Treatment of graft rejection following solid organ transplantation Action: For adoption Documents tabled: Draft Summary report

2.2.22.

- EMA/OD/253/16 Treatment of invasive aspergillosis Action: For adoption Documents tabled: Draft Summary report Notes:

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There have been 2 designations for this condition: EMA/OD/009/14 Isavuconazonium sulfate, EMA/OD/104/16 2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)- N-{4-[4-(5-fluoropyrimidin-2-yl) piperazin- 1-yl]-phenyl}-2-oxo-acetamide

2.3.

Revision of the COMP opinions None

2.4.

Amendment of existing orphan designations None

2.5.

Appeal

2.5.1.

20% I.V. fat emulsion consisting of 20% soybean oil, 1.2% egg yolk phospholipids, 2.25% glycerin, and water for injection - EMA/OD/062/16 Alan Boyd Consultants Ltd; Treatment of poisoning by local anesthetics Action: For information, Oral explanation scheduled in March 2017 Documents tabled: Grounds for appeal Notes: Appeal of the negative COMP opinion adopted in November 2016.

2.6.

Nominations

2.6.1.

New applications for orphan medicinal product designation - Appointment of COMP coordinators Action: For adoption Document tabled: OMPD applications - appointment of coord. at the 14-16 February 2017 COMP meeting

2.7.

Evaluation on-going Seven applications for orphan designation will not be discussed as evaluation is on-going. Action: For information Notes: See 7.8.1. Table 6. Evaluation Ongoing.

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3.

Requests for protocol assistance with significant benefit question

3.1.

Ongoing procedures

3.1.1.

Treatment of Gaucher disease Action: For discussion

3.1.2.

Treatment of amyotrophic lateral sclerosis Action: For adoption

3.1.3.

Treatment of narcolepsy Action: For adoption

3.1.4.

Treatment of Langerhans cell histiocytosis Action: For discussion

3.1.5.

Treatment of glioma Action: For adoption

3.1.6.

Treatment of Wolfram syndrome Action: For discussion

3.1.7.

Treatment of Wolfram syndrome Action: For discussion

3.2. 3.2.1.

Finalised letters Treatment of paroxysmal nocturnal haemoglobinuria

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Action: For information

3.2.2.

Treatment of autosomal dominant polycystic kidney disease Action: For information

3.3. 3.3.1.

New requests Treatment of paroxysmal nocturnal haemoglobinuria Action: For information

4.

Review of orphan designation for orphan medicinal products for marketing authorisation

4.1.

Orphan designated products for which CHMP opinions have been adopted None

4.2. 4.2.1.

Orphan designated products for discussion prior to adoption of CHMP opinion - cerliponase alfa - EMA/OD/177/12, EU/3/13/1118, EMEA/H/C/004065 BioMarin International Limited; Treatment of neuronal ceroid lipofuscinosis type 2 Action: For discussion Document(s) tabled: Draft report on review of OMPD

4.2.2.

- parathyroid hormone – EMA/OD/102/13, EU/3/13/1210, EMEA/H/C/003861 NPS Pharma Holdings Limited; Treatment of hypoparathyroidism Action: For discussion Document(s) tabled: Draft report on review of OMPD

4.2.3.

- pentosan polysulfate sodium – EMA/OD/179/14, EU/3/14/1411, EMEA/H/C/004246 Bene-Arzneimittel GmbH; Treatment of interstitial cystitis Action: For discussion, Oral explanation postponed to March 2017 Documents tabled:

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Draft report on review of OMPD CHMP assessment report

4.3.

Appeal None

4.4.

On-going procedures Action: For information

4.5.

Public Summary of Opinions Action: For information

5.

Review of orphan designation for authorised orphan medicinal products at time marketing authorisation extension

5.1.

After adoption of CHMP opinion None

5.2.

Prior to adoption of CHMP opinion None

5.3.

Appeal None

6.

Application of Article 8(2) of the Orphan Regulation None

7.

Organisational, regulatory and methodological matters

7.1.

Mandate and organisation of the COMP

7.1.1.

Protocol Assistance Working Group Proposed meeting time on 14 February 2017 at time 13:00 Document(s) tabled: PAWG draft agenda for February 2017 meeting PAWG draft minutes for January 2017 meeting

7.1.2.

Strategic Review & Learning meetings None

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7.1.3.

Survey to Committee members, alternates and concerned NCA staff on the service and support provided by EMA Committee Secretariats - Findings Action: For information

7.1.4.

Selection procedure for COMP members nominated by the EC on EMA recommendation Action: For information

7.2.

Coordination with EMA Scientific Committees or CMDh-v

7.2.1.

PDCO/COMP Working Group Proposed meeting time on 15 February 2017 at time 13:00

7.2.2.

Recommendations on eligibility to PRIME – report from CHMP Action: For information Document(s) tabled: PRIME eligibility requests - list of adopted outcomes January 2017

7.3.

Coordination with EMA Working Parties/Working Groups/Drafting Groups None

7.4.

Cooperation within the EU regulatory network

7.4.1.

European Commission None

7.5.

Cooperation with International Regulators

7.5.1.

Food and Drug Administration (FDA) Action: For information Document tabled: Draft Agenda January 24, 2017

7.5.2.

Japanese Pharmaceuticals and Medical Devices Agency (PMDA) None

7.5.3.

The Therapeutic Goods Administration (TGA), Australia None

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7.5.4.

Health Canada None

7.6.

Contacts of the COMP with external parties and interaction with the Interested Parties to the Committee None

7.7.

COMP work plan

7.7.1.

COMP Work Plan 2017 Action: For information Document(s) tabled: Updated COMP Work Plan 2017

7.8.

Planning and reporting

7.8.1.

List of all applications submitted/expected and the COMP coordinatorship distribution of valid applications submitted in 2017 Action: For information

7.8.2.

Overview of orphan marketing authorisations/applications Action: For information

8. 8.1.

Any other business Action: For information

9.

Explanatory notes The notes below give a brief explanation of the main sections and headings in the COMP agenda and should be read in conjunction with the agenda or the minutes. Abbreviations / Acronyms CHMP: Committee for Medicinal Product for Human Use COMP: Committee for Orphan Medicinal Products EC: European Commission OD: Orphan Designation PA: Protocol Assistance PDCO: Paediatric Committee PRAC: Pharmacovigilance and Risk Assessment Committee SA: Scientific Advice SAWP: Scientific Advice Working Party

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Orphan Designation (section 2 Applications for orphan medicinal product designation) The orphan designation is the appellation given to certain medicinal products under development that are intended to diagnose, prevent or treat rare conditions when they meet a pre-defined set of criteria foreseen in the legislation. Medicinal products which get the orphan status benefit from several incentives (fee reductions for regulatory procedures (including protocol assistance), national incentives for research and development, 10-year market exclusivity) aiming at stimulating the development and availability of treatments for patients suffering from rare diseases. Orphan Designations are granted by Decisions of the European Commission based on opinions from the COMP. Orphan designated medicinal products are entered in the Community Register of Orphan Medicinal Products. Protocol Assistance (section 3 Requests for protocol assistance with significant benefit question) The protocol assistance is the help provided by the Agency to the sponsor of an orphan medicinal product, on the conduct of the various tests and trials necessary to demonstrate the quality, safety and efficacy of the medicinal product in view of the submission of an application for marketing authorisation. Sponsor Any legal or physical person, established in the Community, seeking to obtain or having obtained the designation of a medicinal product as an orphan medicinal product. Maintenance of Orphan Designation (section 4 Review of orphan designation for orphan medicinal products for marketing authorisation). At the time of marketing authorisation, the COMP will check if all criteria for orphan designation are still met. The designated orphan medicinal product should be removed from the Community Register of Orphan Medicinal Products if it is established that the criteria laid down in the legislation are no longer met. More detailed information on the above terms can be found on the EMA website: www.ema.europa.eu/

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